NCT03364153CompletedPhase 2
Zimura Compared to Sham in Patients With Autosomal Recessive Stargardt Disease (STGD1)
A Phase 2b Randomized, Double-masked, Controlled Trial to Establish the Safety and Efficacy of Zimura™ (Complement C5 Inhibitor) Compared to Sham in Subjects With Autosomal Recessive Stargardt Disease
Source: ClinicalTrials.gov · Last updated 15 Apr 2026
Brief summary (registry, in English)
The purpose of this study is to evaluate the safety and efficacy of avacincaptad pegol intravitreal injection compared to Sham in participants with autosomal recessive Stargardt disease 1 (STGD1).
- Status (registry)
- Completed
- Phase (registry)
- Phase 2
- Study type
- Interventional
- Enrollment
- 121 (actual)
- First submitted
- 1 Dec 2017
- Start date
- 12 Jan 2018
- Primary completion date
- 31 Mar 2025
- Lead sponsor
- Astellas Pharma Global Development, Inc.
- NCT number
- NCT03364153
- EudraCT number
- 2017-004783-35
What you commit to
- Study type: Interventional · Phase 2
- Participants enrolled: 121
- From 12 Jan 2018 to the primary completion date, 31 Mar 2025
- 41 sites in 8 countries
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Any prior treatment for STGD1 including gene therapy, stem cell therapy or any prior intravitreal treatment for any indication in either eye
- Participation in an interventional study of a vitamin A derivative \</= 3 months prior to screening
- Any intraocular surgery or thermal laser within 3 months of trial entry. Any prior thermal laser in the macular region
- Any major surgical procedure within one month of trial entry or anticipated during the trial
- Any treatment with an investigational agent in the past 60 days for any condition
Other illness
- Macular atrophy secondary to any condition other than STGD1 in either eye
- Presence of intraocular inflammation, macular hole, pathologic myopia, epiretinal membrane, evidence of significant vitreo-macular traction, vitreous hemorrhage or aphakia
- Diabetes mellitus
- Stroke within 12 months of trial entry
- Known serious allergies to the fluorescein dye used in angiography, povidone iodine, or to the components of the avacincaptad pegol formulation
Lab values
- Hemoglobin A1c (HbA1c) value of \>/=6.5%
Other
- Women who are pregnant or nursing
Who the study is looking for
- At least two pathogenic mutations of ATP-Binding Cassette (ABC)A4 gene confirmed by a Clinical Laboratory Improvement Amendments (CLIA)-certified laboratory
- Best corrected visual acuity in the study eye between 20/20 - 20/200 Snellen equivalent, inclusive
Age in the registry: 18 years to 60 years · Sex: All · Does not accept healthy volunteers
Sites (41)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| University of Bonn Coordinator of this site? Confirm its status | Bonn | Germany | Unknown status |
| Augenklinik der LMU München Coordinator of this site? Confirm its status | München | Germany | Unknown status |
| University of Tuebingen Coordinator of this site? Confirm its status | Tübingen | Germany | Unknown status |
| Institut de la Macula Coordinator of this site? Confirm its status | Barcelona | Spain | Unknown status |
| Creteil University Eye Clinic University Paris EST Coordinator of this site? Confirm its status | Créteil | France | Unknown status |
| Hôpital de la Croix Rousse Coordinator of this site? Confirm its status | Lyon | France | Unknown status |
| Centre ophtalmologique des Quinzes Vingts Coordinator of this site? Confirm its status | Paris | France | Unknown status |
| Princess Alexandra Eye Pavillion Coordinator of this site? Confirm its status | Edinburgh | United Kingdom | Unknown status |
| Moorfields Eye Hospital Coordinator of this site? Confirm its status | London | United Kingdom | Unknown status |
| Budapest Retina Institute Coordinator of this site? Confirm its status | Budapest | Hungary | Unknown status |
| Semmelweis Egyetem Coordinator of this site? Confirm its status | Budapest | Hungary | Unknown status |
| University of Debrecen DE KK Szemészeti Klinika Coordinator of this site? Confirm its status | Debrecen | Hungary | Unknown status |
| Ganglion Medical Center Coordinator of this site? Confirm its status | Pécs | Hungary | Unknown status |
| Szegedi Tudományegyetem Szent-Györgyi Albert Klinikai Központ, Szemészeti Klinika Coordinator of this site? Confirm its status | Szeged | Hungary | Unknown status |
| Rambam Health Care Campus Coordinator of this site? Confirm its status | Haifa | Israel | Unknown status |
| Hadassah University Hospital Coordinator of this site? Confirm its status | Jerusalem | Israel | Unknown status |
| Rabin Medical Center-Beilinson Campus Coordinator of this site? Confirm its status | Petah Tikva | Israel | Unknown status |
| Kaplan Medical Center Coordinator of this site? Confirm its status | Rehovot | Israel | Unknown status |
| Tel Aviv Sourasky Medical Center (Ichilov Hospital) Coordinator of this site? Confirm its status | Tel Aviv | Israel | Unknown status |
| AOU Policlinico Sant'Orsola Malpighi, U.O. Oftalmologia, Coordinator of this site? Confirm its status | Bologna | Italy | Unknown status |
| Azienda Ospedaliera Universitaria Careggi Coordinator of this site? Confirm its status | Florence | Italy | Unknown status |
| Ospedale San Raffaele Coordinator of this site? Confirm its status | Milan | Italy | Unknown status |
| University of Campania Luigi Vanvitelli Eye Clinic Coordinator of this site? Confirm its status | Naples | Italy | Unknown status |
| Fondazione Policlinico Tor Vergata, UOSD Patologie Retiniche Coordinator of this site? Confirm its status | Rome | Italy | Unknown status |
| University of Michigan Kellogg Eye Center Coordinator of this site? Confirm its status | Ann Arbor | United States | Unknown status |
| Austin Retina Associates Coordinator of this site? Confirm its status | Austin | United States | Unknown status |
| Wilmer Eye Institute Johns Hopkins Coordinator of this site? Confirm its status | Baltimore | United States | Unknown status |
| Retina Center of NJ, LLC. Coordinator of this site? Confirm its status | Bloomfield | United States | Unknown status |
| Ophthalmic Consultants of Boston Coordinator of this site? Confirm its status | Boston | United States | Unknown status |
| Retina Foundation of the Southwest Coordinator of this site? Confirm its status | Dallas | United States | Unknown status |
| VitreoRetinal Associates Coordinator of this site? Confirm its status | Gainesville | United States | Unknown status |
| Jules Stein Eye Institute/ David Geffen School of Medicine Coordinator of this site? Confirm its status | Los Angeles | United States | Unknown status |
| The Retina Center Coordinator of this site? Confirm its status | Minneapolis | United States | Unknown status |
| Retina Specialty Institute Coordinator of this site? Confirm its status | Pensacola | United States | Unknown status |
| Wills Eye Hospital/Mid Atlantic Retina Coordinator of this site? Confirm its status | Philadelphia | United States | Unknown status |
| Retinal Research Institute Coordinator of this site? Confirm its status | Phoenix | United States | Unknown status |
| UPMC Eye Center Coordinator of this site? Confirm its status | Pittsburgh | United States | Unknown status |
| Casey Eye Institute, Oregon Health & Science University Coordinator of this site? Confirm its status | Portland | United States | Unknown status |
| University of Utah John A. Moran Eye Center Coordinator of this site? Confirm its status | Salt Lake City | United States | Unknown status |
| Palmetto Retina Center Coordinator of this site? Confirm its status | West Columbia | United States | Unknown status |
| Strategic Clinical Research Group Coordinator of this site? Confirm its status | Willow Park | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.